orphan drugs understanding the rare disease market and its dynamics woodhead publishing series in biomedicine

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Orphan Drugs

Author : Elizabeth Hernberg-Ståhl
ISBN : 9781908818393
Genre : Medical
File Size : 66. 23 MB
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This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups. The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey. After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs. The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs. A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patients A source of updated information, news and trends for those who are already active in this fast-evolving field Covers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe

A Practical Guide To Rational Drug Design

Author : Sun Hongmao
ISBN : 9780081001059
Genre : Medical
File Size : 68. 22 MB
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This book is not going to be an exhaustive survey covering all aspects of rational drug design. Instead, it is going to provide critical know-how through real-world examples. Relevant case studies will be presented and analyzed to illustrate the following: how to optimize a lead compound whether one has high or low levels of structural information; how to derive hits from competitors’ active compounds or from natural ligands of the targets; how to springboard from competitors’ SAR knowledge in lead optimization; how to design a ligand to interfere with protein-protein interactions by correctly examining the PPI interface; how to circumvent IP blockage using data mining; how to construct and fully utilize a knowledge-based molecular descriptor system; how to build a reliable QSAR model by focusing on data quality and proper selection of molecular descriptors and statistical approaches. A Practical Guide to Rational Drug Design focuses on computational drug design, with only basic coverage of biology and chemistry issues, such as assay design, target validation and synthetic routes. Discusses various tactics applicable to daily drug design Readers can download the materials used in the book, including structures, scripts, raw data, protocols, and codes, making this book suitable resource for short courses or workshops Offers a unique viewpoint on drug discovery research due to the author’s cross-discipline education background Explores the author’s rich experiences in both pharmaceutical and academic settings

Therapeutic Risk Management Of Medicines

Author : Stephen J. Mayall
ISBN : 9781908818270
Genre : Medical
File Size : 56. 5 MB
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Therapeutic risk management of medicines is an authoritative and practical guide on developing, implementing and evaluating risk management plans for medicines globally. It explains how to assess risks and benefit-risk balance, design and roll out risk minimisation and pharmacovigilance activities, and interact effectively with key stakeholders. A more systematic approach for managing the risks of medicines arose following a number of high-profile drug safety incidents and a need for better access to effective but potentially risky treatments. Regulatory requirements have evolved rapidly over the past decade. Risk management plans (RMPs) are mandatory for new medicinal products in the EU and a Risk Evaluation and Mitigation Strategy (REMS) is needed for certain drugs in the US. This book is an easy-to-read resource that complements current regulatory guidance, by exploring key areas and practical implications in greater detail. It is structured into chapters encompassing a background to therapeutic risk management, strategies for developing RMPs, implementation of RMPs, and the continuing evolution of the risk management field.The topic is of critical importance not only to the pharmaceutical and biotechnology industries, but also regulators and healthcare policymakers.Some chapters feature contributions from selected industry experts. An up-to-date practical guide on conceiving, designing, and implementing global therapeutic risk management plans for medicines A number of useful frameworks are presented which add impact to RMPs (Risk Management Plans), together with regional specific information (European Union, United States, and Japan) A comprehensive guide for performing risk management more effectively throughout a product’s life-cycle

Procli In Platonis Parmenidem Commentaria Libros Iv V Continens

Author : Proclus
ISBN : STANFORD:36105217831093
Genre : Literary Collections
File Size : 47. 79 MB
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The Commentary on Plato's Parmenides by Proclus (AD 412-85) is the most important extant document on the interpretation of this enigmatic dialogue, and has had a crucial influence on all subsequent readings. In Proclus' Commentary, the Parmenides provides the argumentative and conceptual framework for a scientific theology wherein all mythological discourse about the gods can be integrated. Its exposition was therefore the culmination of the curriculum of the Platonic school. This theological reading of the Parmenides persisted, through the medium of Ficino, until the nineteenth century. Previously this important text was only accessible in the edition of V. Cousin (Paris, 1864). This new critical edition is based on an exhaustive study of both the Greek tradition and the medieval Latin translation. This volume contains Books IV and V.

Oxford English Greek Learner S Dictionary

Author : Д. Н Ставропулос
ISBN : 0194312429
Genre : English language
File Size : 74. 95 MB
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This dictionary provides definitions and common usage with numerous example sentences and phrases.

Collectio Vaticana 1 32

Author : Eduard Schwartz
ISBN : 9783110822472
Genre : Religion
File Size : 79. 50 MB
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Acta Conciliorum Oecumenicorum Iussu Atque Mandato Societatis Scientiarum Argentoratensis Tom I Concilium Universale Ephesenum Vol I Acta Graeca Pars Vi Collectio Vaticana 165 172

Author : Eduard Schwartz
ISBN : 3110004097
Genre : Church history
File Size : 43. 50 MB
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